
Sensorion Announces its Participation in the American Society of Cell and Gene Therapy (ASGCT) Annual Meeting
MONTPELLIER, France--(BUSINESS WIRE)--Regulatory News:
Sensorion (FR0012596468 – ALSEN) a pioneering clinical-stage biotechnology company specializing in the development of novel therapies to restore, treat and prevent hearing loss disorders, today announced its participation at the 28 th Annual American Society of Cell and Gene Therapy Meeting (ASGCT), taking place on May 13-17, 2025, in New-Orleans, United States of America.
On this occasion, Rafik Boudra, Preclinical Group Leader Technology & Innovation Platform at Sensorion, will present the 2 following posters on May 15, 2025:
Poster N°1556
Title: Safety and efficacy of GJB2-GT, an adeno associated viral vector-based gene therapy treatment candidate for the autosomal recessive non-syndromic deafness 1A (DFNB1A)
Poster N° 1559
Title: GJB2 gene therapy-response of two pre-clinical mouse models of the most frequent form of human deafness, DFNB1A
This poster is presented in collaboration with the Auditory Therapies Innovation Laboratory headed by Professor Christine PETIT at Hearing Institute, research center of the Institut Pasteur
Date: May 15, 2025
Time: 5.30 pm - 7.00 pm CT
Room: Morial Convention Center, Poster Hall, Hall I2
About Sensorion
Sensorion is a pioneering clinical-stage biotech company, which specializes in the development of novel therapies to restore, treat, and prevent hearing loss disorders, a significant global unmet medical need. Sensorion has built a unique R&D technology platform to expand its understanding of the pathophysiology and etiology of inner ear related diseases, enabling it to select the best targets and mechanisms of action for drug candidates.
It has two gene therapy programs aimed at correcting hereditary monogenic forms of deafness, developed in the framework of its broad strategic collaboration focused on the genetics of hearing with the Institut Pasteur. SENS-501 (OTOF-GT) currently being developed in a Phase 1/2 clinical trial, targets deafness caused by mutations of the gene encoding for otoferlin and GJB2-GT targets hearing loss related to mutations in GJB2 gene to potentially address important hearing loss segments in adults and children. The Company is also working on the identification of biomarkers to improve diagnosis of these underserved illnesses.
Sensorion's portfolio also comprises programs of a clinical-stage small molecule, SENS-401 (Arazasetron), for the treatment and prevention of hearing loss disorders. Sensorion's small molecule progresses in a Phase 2 proof of concept clinical study of SENS-401 in Cisplatin-Induced Ototoxicity (CIO) for the preservation of residual hearing. Sensorion, with partner Cochlear Limited, completed in 2024 a Phase 2a study of SENS-401 for the residual hearing preservation in patients scheduled for cochlear implantation. A Phase 2 study of SENS-401 was also completed in Sudden Sensorineural Hearing Loss (SSNHL) in January 2022.
www.sensorion.com
Disclaimer
This press release contains certain forward-looking statements concerning Sensorion and its business. Such forward looking statements are based on assumptions that Sensorion considers to be reasonable. However, there can be no assurance that such forward-looking statements will be verified, which statements are subject to numerous risks, including the risks set forth in the 2024 full year report published on March 14, 2025, and available on our website and to the development of economic conditions, financial markets and the markets in which Sensorion operates. The forward-looking statements contained in this press release are also subject to risks not yet known to Sensorion or not currently considered material by Sensorion. The occurrence of all or part of such risks could cause actual results, financial conditions, performance or achievements of Sensorion to be materially different from such forward-looking statements. This press release and the information that it contains do not constitute an offer to sell or subscribe for, or a solicitation of an offer to purchase or subscribe for, Sensorion shares in any country. The communication of this press release in certain countries may constitute a violation of local laws and regulations. Any recipient of this press release must inform oneself of any such local restrictions and comply therewith.
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Those risks and uncertainties include, among other things, the risks listed under the heading "Risk Factors" in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at and available through the company's website at You should not place undue reliance on these statements, or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. (VRTX-GEN) View source version on Contacts Vertex Pharmaceuticals Incorporated Investors: InvestorInfo@ Media: mediainfo@ orInternational: +44 20 3204 5275 Sign in to access your portfolio


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BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced data across multiple studies demonstrating positive clinical and quality of life benefits of treatment with CFTR modulators and, in particular, ALYFTREK ® (vanzacaftor/tezacaftor/deutivacaftor), which is approved in the United States and United Kingdom and is currently under review with health authorities in the EU, Canada, Australia, New Zealand and Switzerland. These data were presented at this year's European Cystic Fibrosis Society's (ECFS) 48th European Cystic Fibrosis Conference held June 4-7, 2025, in Milan, Italy. During the conference, the Company presented data from a pooled analysis across CFTR modulators, including ALYFTREK, which demonstrate that reduction in sweat chloride (SwCl), and therefore greater restoration of CFTR function, is associated with improved outcomes in people with cystic fibrosis (CF). For all clinical outcomes in the study, SwCl levels below 60 mmol/L were associated with greater benefit including better and more stable lung function, fewer pulmonary exacerbations, better nutritional status and better quality of life. SwCl levels below 30 mmol/L generally demonstrated greater numerical benefit than all other groups, with confidence intervals that overlapped with the ≥30 to <60 mmol/L group. Vertex also presented the results of a post hoc analysis from the Phase 3 randomized, controlled and open-label trials of ALYFTREK (abstract WS19.04) which suggest treatment with ALYFTREK is associated with improved health-related quality of life outcomes in adolescents and adults, and with improved CF symptoms and general functioning in children aged 6-11 years compared to patients treated with TRIKAFTA. 'These new data further demonstrate that reducing sweat chloride via treatment with CFTR modulators drives improved CFTR function and may ultimately result in better outcomes for patients across a variety of measures,' said Professor Isabelle Fajac, Pulmonologist, Professor of Physiology at APHP-Université Paris Cité, Paris, France. 'Importantly, the data also indicate that ALYFTREK, which has been shown to deliver even greater reductions in sweat chloride than TRIKAFTA, may drive improved quality of life and other health-related outcomes above and beyond even what we've seen with CFTR modulators to date.' About Cystic Fibrosis Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 109,000 people, including 94,000 people in North America, Europe and Australia. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. 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Today Vertex CF medicines are treating over 75,000 people with CF in more than 60 countries on six continents. This represents approximately 2/3 of the diagnosed people with CF eligible for CFTR modulator therapy. In addition, we have established a pilot donation program in collaboration with Direct Relief to provide TRIKAFTA® to eligible people with CF in select lower-income countries. The program currently includes 14 countries across four continents. 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The dose of ALYFTREK or TRIKAFTA should be reduced when used concomitantly with moderate or strong CYP3A inhibitors Use With CYP3A Inducers Following concomitant use of strong or moderate CYP3A inducers with ALYFTREK, exposures of vanzacaftor, tezacaftor, and deutivacaftor were decreased, which may reduce ALYFTREK effectiveness. Concomitant use with strong or moderate CYP3A inducers is not recommended Exposure to ivacaftor is significantly decreased and exposure to elexacaftor and tezacaftor are expected to decrease by concomitant use with CYP3A inducers, which may reduce therapeutic effectiveness of TRIKAFTA. Concomitant use with strong CYP3A inducers, such as rifampin, rifabutin, phenobarbital, carbamazepine, phenytoin, and St. John's wort, is not recommended CATARACTS Non-congenital lens opacities/cataracts have been reported in pediatric patients treated with TRIKAFTA, which contain ivacaftor (similar to an active ingredient in ALYFTREK). Baseline and follow-up ophthalmological examinations are recommended in pediatric patients initiating treatment ADVERSE REACTIONS ALYFTREK Serious adverse reactions that occurred more frequently with ALYFTREK than with ELX/TEZ/IVA in 2 or more patients (≥0.4%) were influenza (1.5%), increased AST (0.4%), increased GGT (0.4%), depression (0.4%), and syncope (0.4%) The most common adverse reactions occurring in ≥5% of patients and at a frequency higher than ELX/TEZ/IVA by ≥1% were cough, nasopharyngitis, upper respiratory tract infection, headache, oropharyngeal pain, influenza, fatigue, increased ALT, rash, increased AST, and sinus congestion TRIKAFTA Serious adverse reactions that occurred more frequently in patients treated with TRIKAFTA compared to placebo included rash (1% vs <1%) and influenza (1% vs 0%) The most common adverse reactions occurring in ≥5% of patients treated with TRIKAFTA and at a rate higher than placebo by ≥1% were headache, upper respiratory tract infection, abdominal pain, diarrhea, rash, alanine aminotransferase increased, nasal congestion, blood creatine phosphokinase increased, aspartate aminotransferase increased, rhinorrhea, rhinitis, influenza, sinusitis, blood bilirubin increased, and constipation USE IN SPECIFIC POPULATIONS PEDIATRIC USE Safety and effectiveness have not been established for ALYFTREK in patients younger than 6 years of age, nor for TRIKAFTA in patients younger than 2 years of age. The use in children under these ages is not recommended Please see full Prescribing Information, including Boxed Warning, for Alfytrek and Trikafta. About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 15 consecutive years on Science magazine's Top Employers list and one of Fortune's 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit or follow us on LinkedIn, Facebook, Instagram, YouTube and X. Special Note Regarding Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements by Professor Isabelle Fajac, in this press release, and statements regarding Vertex's beliefs about the potential benefits of ALYFTREK and TRIKAFTA for people with CF, and expectations for the pilot donation program. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, the risks listed under the heading 'Risk Factors' in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at and available through the company's website at You should not place undue reliance on these statements, or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. (VRTX-GEN)